The Week in Review
With Congress on recess, news developments in Washington were limited to the White House. On Wednesday, President Trump announced via Twitter that he would be replacing Veterans’ Affairs (VA) Secretary David Shulkin with Ronnie Jackson, the White House’s presidential physician. Shulkin – an Obama appointee who was asked to stay on with the Trump Administration – released a statement after his dismissal, in addition to several on camera interviews, suggesting that the White House wanted to replace him in order to advance efforts to privatize the VA. Defense Department official Robert Wilkie will serve as Acting Secretary until Jackson’s nomination is considered by the Senate.
Also of note, Thorn Run Partners client, Villanova University, advanced to the NCAA Men’s Basketball Championship game with a 95-79 thumping of Kansas on Saturday. They’ll face Michigan, a 69-57 winner over Loyola of Chicago, in the Final tonight in San Antonio.
The Week Ahead
Both the House and Senate will be out for the second week of a two-week break for the Easter and Passover holidays. The Senate is due to return to Washington on Monday, Apr. 8, and the House a day later on Apr. 9.
FDA Announces Plan to Increase Utilization of Patient Experience Data in Benefit-Risk Assessments
The Food and Drug Administration (FDA) has released a new plan, titled “Benefit-Risk Assessment in Drug Regulatory Decision-Making,” which outlines the steps the FDA has taken to enhance benefit-risk assessment in drug review and lays the foundation for future guidance on incorporating patient experience data. Specifically, the document provides a roadmap for enhancing FDA’s Benefit-Risk Framework, working toward a goal of providing new guidance by June 2020 that articulates the FDA’s decision-making context and framework for benefit-risk assessment. This forthcoming guidance will outline how patient experience data and related information can be used to inform benefit-risk assessment.
In a statement announcing the plan, FDA Commissioner Scott Gottlieb suggested that patient feedback should play more of a role in informing the agency’s benefit-risk assessments. “Patients are teaching us about the benefits that matter most to them and the risks that they are most concerned about,” he said. “Patient perception of the benefits and risks of different treatment options can vary based on the stage of the disease, the age of onset, alternative therapies available to treat the disease (if any) and whether a novel therapy improves a patient’s ability to function normally, slows the rate of disease progression or impacts other aspects of a patient’s quality of life.”
In 2009, the FDA began work to develop a structured approach to conducting benefit-risk assessments as part of an effort to bring more clarity and consistency to its regulatory decisions. Under the fifth iteration of the Prescription Drug User Fee Act (PDUFA V), FDA committed to developing a five-year plan to implement a structured benefit-risk assessment in its review processes, and in 2013, the agency released a draft implementation plan describing its approach. In general, the structured benefit-risk assessment looks at information about the condition a drug is meant to treat, currently available treatments, benefits, risks and risk management. FDA then weighs the evidence and uncertainties in each area to inform its conclusions and rationale behind regulatory decisions.
With respect to their methodology, Commissioner Gottlieb noted that FDA has conducted patient-focused drug development meetings in over 20 disease areas, and concluded that patient input can: (1) inform the clinical context and provide insights to frame the assessment of benefits and risk; and (2) provide a direct source of evidence regarding the benefits and risks, if methodologically-sound data collection tools could be developed and used within clinical studies of an investigational therapy. The FDA is now developing guidance to enable more widespread development of such patient experience data to inform regulatory decision-making, as part of their implementation of PDUFA VI and the 21st Century Cures Act.
Senate HELP Chairman Releases FDA-Focused Opioid Proposals
Last Tuesday, Senate Health, Education, Labor and Pensions (HELP) Committee Chairman Lamar Alexander (R-TN) released two discussion draft proposals related to the opioid crisis aimed at strengthening the authority of the Food and Drug Administration (FDA). This builds on the series of bipartisan hearings the Committee has convened since October 2017. It also comes on the heels of a House Energy and Commerce Subcommittee hearing which featured discussion about limitations in FDA’s authority to address certain issues contributing to the opioids crisis. The draft proposals represent the latest developments in the Committee’s work to craft a legislative package focused on the opioids crisis. It may signal they are making progress to solidify the scope of such a package.
Several Members of Congress remain committed to taking additional steps this year to address the opioids and substance abuse crisis — beyond the provisions included in the 2018 omnibus spending bill. The Senate HELP Committee expects to reconvene on this issue sometime this Spring as does the Senate Finance Committee, though the specific timing hinges at least in part on securing sufficient member buy-in on the scope of the packages for consideration. On the other side of the Capitol, the Energy and Commerce Committee has laid out a relatively more specific timeframe. The Subcommittee is preparing for a third hearing on the opioid crisis in April, which will cover Medicare, Medicaid, and payer issues and plans. Committee Chairman Greg Walden (R-OR) has said he wants the Committee to complete consideration of bills to address the opioid crisis by Memorial Day.
Among the highlights of the legislation released by Sen. Alexander:
- Blister Packs and Leftover Drugs — The purpose of this draft language is two-fold. Firstly, it would help limit overprescribing by giving the FDA the authority to require drug manufacturers to package certain opioids in blister packs, allowing for a set dose — for example, a 7 day supply. Secondly, it seeks to prevent inappropriate use of unneeded or unused opioids by encouraging manufactures to provide a safe way to dispose of leftover drugs along with the packaging.
- FDA Coordination With Customs — The draft bill includes several provisions designed to improve coordination between the FDA and Customs Border Protection (CBP) with the goal of finding and seizing illegal drugs at the border.
CMS Finalizes Update to Decades Old Medicaid Drug Rebate Agreement
On March 23, the Centers for Medicare and Medicaid Services (CMS) announced changes (Federal Register) to the Medicaid Program’s National Drug Rebate Agreement (NDRA). This final notice updates the NDRA to incorporate regulatory and legislative changes that have occurred since it was first published in 1991. CMS is also updating the NDRA to make editorial and structural revisions, such as references to the updated Office of Management and Budget (OMB)-approved data collection forms and electronic data reporting. The updated NDRA became applicable on March 23, 2018. Although no new policy changes appear to have been made, manufacturers will need to take specific actions listed in the notice.
CMS asked that written notice with good cause to terminate all old rebate agreements be given at least six months after the effective date of the rule, and that the updated NDRA be signed by all new and participating manufacturers. CMS directed participating manufacturers to work with CMS to sign new NDRAs for each labeler code. The agency promised further guidance will come on how manufacturers can effectively update their NDRAs. This notice did not appear to directly address the intersection of value-based contracting and the Medicaid Drug Rebate Program; however the Administration is expected to address this issue in forthcoming announcements.
Former CVS Executive Nominated to Head HHS Drug Pricing Policy
Former CVS Caremark executive Daniel Best was appointed by Health and Human Services Secretary Alex Azar to lead the department’s initiatives to lower drug prices last week. Daniel Best, whose official title will be Senior Advisor to the Secretary for Drug Pricing Reform, also worked for drug manufacturer Pfizer for 12 years before his time at CVS. At CVS, he was responsible for the company’s prescription drug plans and Medicare Part D plans. It wasn’t immediately clear what initiatives Mr. Best will head.
Sec. Azar also named Brett Giroir Senior Advisor to the Secretary for Mental Health and Opioid Policy, and will be responsible for coordinating HHS’s efforts across the administration to fight the opioid crisis. Giroir and Best are two of four hires Azar expects to make in the coming weeks to lead different priority initiatives: combating the opioid crisis, bringing down the high cost of prescription drugs, addressing the cost and availability of health insurance and shifting to a value-based health system.
Report Shows Prices for Medicare Part D Drugs Rose 12 Percent Annually
A new report released last week by Sen. Claire McCaskill (D-MO) showed that prices for each of the 20 most prescribed branded drugs through the Medicare Part D program have increased in price every year for the last five years. The drugs increased by an average of 12 percent each year, which the senator notes is nearly ten times higher than the rate of inflation, and 12 of the drugs rose in price by more than 50 percent in the five-year period. Six of the 20 drugs had prices increases of over 100 percent. At least one drug manufacturer, Pfizer, has already come out against the report, claiming that it misrepresents the actual prices of the drugs studied. Additionally, the report found that while prescriptions for top brand-name drugs dropped by 48 million, revenue increased by $8.5 billion over the last five years.